Cell and Gene Therapies: Overcoming Barriers to Realize Transformative Potential

Over the past decade, cell and gene therapies (CGTs) have evolved from a theoretical approach to disease treatment into one of health care's most important innovations, offering new excitement and significant potential to treat—and in some cases cure—a growing number of serious diseases. Today, more than 30 CGTs are FDA approved, and experts predict we could see another 10 to 20 approvals annually in the next few years.¹

3d rendering of genes and cells | Image credit: © Anusorn | stock.adobe.com

Although the potential for these groundbreaking therapies to transform care is exciting, significant barriers prevent many patients from accessing advanced care. To better understand these challenges and strategies for overcoming them, InspiroGene by McKesson recently published its 2024 Cell and Gene Therapy Report, which includes interviews with experts across the CGT industry and a survey of 124 US oncologists about their experiences with and attitudes toward CGTs. These insights are the start of an important industry-wide conversation about how all stakeholders can collaborate to expand access for patients.¹

Optimism Tempered by Practical Realities

The survey revealed overwhelming optimism about CGTs among oncologists, with 99% agreeing they are among the most important medical innovations of our time. Additionally, 97% expressed optimism about their potential benefits for patients, including the possibility of a cure.¹

However, this enthusiasm is tempered by practical concerns. Three out of 5 oncologists surveyed say CGTs are "still largely unproven," and more than half (54%) cited concerns about safety profiles as an influential factor in their decision not to refer or treat patients with CGTs. Additionally, 53% mentioned concerns about adverse effect profiles, emphasizing the need for additional clinical data.¹

Limited Access Remains a Key Challenge

Accessibility remains a critical issue, with 64% of oncologists agreeing that CGTs are not easily accessible for patients who meet the labeled indications. In addition, 3 out of 5 physicians say patients they refer for CGTs often receive other treatments instead. When asked why, they cite insurance coverage and out-of-pocket costs as the most common reasons.¹

Geography also plays a role, as most CGT sites of care are concentrated in major metropolitan areas. As many as 60 million Americans live in rural areas, with limited access to CGT-qualified treatment centers, and a recent study noted that the likelihood of receiving a CAR T-cell therapy was reduced by 40% when patients lived 2 to 4 hours from their nearest treatment center.²

Patient Awareness and Education Are Lacking

Patient awareness and understanding of CGTs remain low, with almost half of oncologists in the survey (47%) reporting patients are rarely or almost never aware of these treatment options for their condition. When informed, many patients express concerns about safety, most often describing the therapies as “too new” or “experimental or risky,” according to oncologists. In addition, two-thirds of oncologists (66%) said one of the top reasons they decide not to prescribe a CGT is because “the patient doesn’t want it,” which could indicate a lack of understanding given the high efficacy of these therapies.¹ These perceptions, coupled with the complex and expensive nature of CGT medicines, often deter patients from pursuing treatment.

Overcoming the barriers highlighted in the survey is critical to ensuring these life-saving therapies reach patients. However, there is no simple fix to these challenges. Driving the necessary change will require collaboration and partnership among all the stakeholders in the CGT ecosystem, including manufacturers, health systems, payers, physicians and pharmacists. Below are some of the key actions needed to drive broader access.

Take Steps to Broaden Clinical Confidence

Given that cell and gene therapy is still a nascent market and many of the approved products have only been available for a short time, it is not surprising that many clinicians cite concerns about safety, durability and side effects as reasons they don’t prescribe. As the field evolves, a growing body of real-world data will be critical to expanding clinicians’ confidence in patient outcomes.

Another key to building confidence in the quality and safety of CGTs is standardizing guidelines where possible, for everything from manufacturing to transportation. Several organizations are currently working on developing these standards, including the American Society for Transplantation and Cellular Therapy, which launched an 80/20 task force to standardize 80% of manufacturing requirements.³

In the pharmacy space, the Association for the Advancement of Blood & Biotherapies (AABB) has taken the lead in developing draft guidance on standards for pharmacy CGT handling. As pharmacies play an important role in the cross-functional treatment team, the AABB guidelines are intended to provide process and safety guidance and quality measures. The proposed standards include guidance on the receipt, storage, handling and dispensing of CGTs.⁴ Given the rapid rate of innovation in CGT, initiatives to set standards tailored for these treatments are crucial.

Increase Patient Access by Expanding the CGT Sites of Care

Building wider access to CGTs will be critically important as therapies for broader disease indications such as solid tumors, cardiovascular, autoimmune conditions, and diabetes begin coming to market. Today, care is concentrated at academic medical centers in major metropolitan areas, but as care options expand, moving more CGTs into community hospital and clinic settings will increase capacity for treating larger patient populations.

A hub-and-spoke model in which academic medical centers collaborate with community sites could help facilitate this expansion. In this framework, large academic medical centers (hubs) focus on delivering complex, cutting-edge treatments and conducting early-phase clinical trials. These centers then work with community hospitals and clinics (spokes) to administer more established therapies. This setup allows patients to receive advanced treatments closer to home, reducing the travel burden and improving access for those in rural or underserved areas.⁵

Expanded training in CGT care will also be necessary to support greater access, not just for physicians, but also for nurses, pharmacists, and care coordinators. In fact, the InspiroGene survey showed that more than half of oncologists (53%) see expanded clinical training as a key priority for expanding patient access. Colleges and universities cannot do this alone; collaboration with hospitals, industry associations, manufacturers, and state agencies will be needed.

Provide Education to Drive Broader Patient Awareness

Beyond expanding training for CGT providers and support staff, the health care industry should educate patients more broadly on the potential benefits and risks these medicines provide. Clinicians and pharmacists will play a more active role in educating their patients, but they will also need support from manufacturers and advocacy groups. Building awareness will become more important, especially as CGTs expand into broader disease categories.

The Path Forward

Although significant hurdles remain, the potential for CGTs to transform patient care is undeniable. As more therapies gain approval and expand into new disease areas, pharmacists, along with other providers, will play an increasingly critical role on the treatment team in patient education and the handling and administration of these complex treatments.

By working together to address current barriers, stakeholders across health care can help realize the transformative potential of cell and gene therapies, bringing new hope to patients facing serious diseases.

REFERENCES

1. 2024 Cell and Gene Therapy Report. InspiroGene By McKesson. 2024. Accessed December 2, 2024. https://inspirogene.com/2024cgtreport/

2. Ahmed N, Sun F, Teigland C, et al. Chimeric antigen receptor T-cell access in patients with relapsed/refractory large B-cell lymphoma: association of access with social determinants of health and travel time to treatment centers. Transp Cell Ther. 2024;30(7):714-725. doi:10.1016/j.jtct.2024.04.017

3. Nikiforow S, Frigault MJ, Frey NV, et al. Paving the road for chimeric antigen receptor T cells: American Society for Transplantation and Cellular Therapy 80/20 Task Force consensus on challenges and solutions to improving efficiency of clinical center certification and maintenance operations for commercially approved immune effector cell therapies. Transplant Cell Ther. 2023;29(4):228-239. doi:10.1016/j.jtct.2023.01.021

4. Proposed 1st Edition of Cell and Gene Therapy Standards for Pharmacy. Association for the Advancement of Blood & Biotherapies. October 16, 2024. Accessed December 2, 2024. https://www.aabb.org/docs/default-source/default-document-library/standards/proposed-1st-first-edition-of-cell-and-gene-therapy-standards-for-pharmacy.pdf?sfvrsn=207365cd_3

5. Saleh S, Dabbous O, Sullivans SD, et al. A practical approach for adoption of a hub and spoke model for cell and gene therapies in low- and middle-income countries: framework and case studies. Gene Ther. 2023;31(1-2):1-11. doi:10.1038/s41434-023-00425-x